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IPOPI PID Patient Needs & Outlooks Survey
A Report based on 300 patient questionnaires
Report Prepared by
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PID Patient Needs & Outlooks Study
Background and objectives 3
Research methodology 4
I. Treatment: i. Background 5
I. Treatment: ii. Evaluation of current treatment 16
II. Living with PID 24
III. Future treatment needs 30
IV. General Health: EQ-5d, SF-12, SF-10 34
V. Information sources 38
VI. Sample profile 43
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Background & objectives Background
• Primary immunodeficiencies (PID) are a group of diseases in which an individual lacks some of the components of the immune system: cells and/or proteins. This makes the individual more susceptible to infections
• The symptoms of PID can range from mild to severe
• PIDs are caused by hereditary or genetic defects in the immune system and WHO has identified over 150 specific types
• The International Patient Organisation for Primary Immunodeficiencies (IPOPI) is the association of national patient organisations and is dedicated to improving awareness, access to early diagnosis and optimal treatments for primary immunodeficiency patients worldwide
Research goals and objectives
• The study has been designed to provide detail on the current landscape, outlook and needs of patients in relation to their circumstances, outlooks and treatment needs with PID
• This study explores the patient experience of PID, covering aspects from treatment and unmet needs to the impact of PID on daily and social life.
• The conjoint section asked respondents to evaluate a number of treatment options in rotation to establish unmet needs.
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Research methodology • Mode: Online (CAWI)
• Duration: 30 minutes
• Fieldwork: 8 April – 17 October 2011
• Sample: N=300: Patients & Care-givers of people with PID and treated with immunoglobulins. Sample sourced through national member organisations (NMOs) affiliated to the International Patient Organisation for Primary Immunodeficiencies (IPOPI). Sample was self-selecting amongst those invited by the NMOs. Questions were phrased differently so as to be appropriate for patients (e.g. “What is your current diagnosis?”) and care-givers (“What is the diagnosis of the PID patient you care for?”)
• Language: Respondents were able to answer the survey in English, French, German, Italian, Spanish or Portuguese
• Countries: Shown in table on right
The research study was sponsored by Baxter and carried out by an independent market research company (BRYTER) in compliance with current codes of conduct and good practice. Privacy and Data Protection: Bryter act in accordance with the ABPI, MRS and BHBIA codes of conduct regarding respondent anonymity and confidentiality: the aim of the market research survey was to gain views and was not promotional. Bryter are fully compliant with the Data Protection Act, so that any information provided by respondents is treated in the strictest confidence: all results are pooled so answers are not attributed to any individuals.
Country Groups Interviews
UK A, B 59
Sweden A 34
Canada C 31
France A, B 31
Germany A, B 31
Spain A, B 22
Portugal A 21
Argentina C 15
Brazil C 13
South Africa 10
Colombia C 9
Italy A, B 9
Switzerland A 4
Belgium A 2
New Zealand 2
Poland A 2
Australia 1
Austria A 1
Hungary A 1
India 1
Netherlands A 1
Groups: A (“Europe”), B (“EU5”), C
(“Americas”), D (“South America”)
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I. Treatment
Background
-
53%
45%
68%
15%
32%
19%
77%
43%
69%
45%
89%
45%
53%
32%
82%
61%
77%
23%
57%
28%
52%
8%
3%
6%
3%
3%
3%
3%
All respondents
Europe
UK
Sweden
France
Germany
Spain*
Portugal*
Americas
Canada
South America
Intravenous Subcutaneous Other
D4. What route of infusion if used to administer your/the patient’s current immunoglobulin therapy? Base: All Respondents (300), Europe (218), UK (59), Sweden (34), France (31), Germany (31), Spain (22), Portugal (21), Americas (68), Canada (31)
Of the sample interviewed, 53% receive intravenous Ig infusions and 45% are infused subcutaneously. Usage of subcutaneous infusion is significantly higher in Europe than in the Americas
CAUTION: *Small base (under 30) ineligible for sig testing
Usage of intravenous administration varies by
countries in Europe.
Intravenous administration in the UK is significantly
higher than Europe average.
In the Americas, overall intravenous administration
used significantly more than in Europe, mainly
driven by South American respondents
Level of subcutaneous administration is
significantly above average in Sweden and Germany.
Route of infusion
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For 42%, immunology specialists are the main decision maker regarding how Ig therapy is administered. However, around 70% of patients and 77% of caregivers were involved in the decision-making process
D5a. Thinking about the decisions and selection of the current therapy… Who was involved in choosing how the therapy is administered? Base: All Respondents (300)
42%
4%
13%
51%
73%
56%
50%
34%
17%
4%
7%
23%
29%
48%
60%
75%
92%
5%
6%
4%
Specialist ImmunologyDoctor
Caregiver
The Patient
Family / Partner
Nurse
Paediatric Doctor
Pharmacist
Single MAIN person deciding Involved / Influential Not involved Don't know
Decision makers for route of administration
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Understandably, the time between infusions is predetermined by the route of administration. The majority of intravenous patients (87%) have at least 3 weeks
between infusions, while 99% subcutaneous patients wait a maximum of a week
D6. Typically, how long do you wait between your current immunoglobulin therapy infusions?
Base: All Respondents (300), Intravenous (160), Subcutaneous (134)
7%
14%
5%
10%
34%
75%
6%
12%
23%
41%
25%
46%
All respondents
Intravenous
Subcutaneous
Less than 3 days 3-4 days A week 2 weeks 3 weeks 4 weeks or moreTime between infusions Average time
between
infusions
16 days
23 days
6 days
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Over 9 in 10 subcutaneous patients are infused at home, while most intravenous patients receive their treatment in
a regional or local hospital (64%) or visit a specialist clinic (11%)
D9. Where does your/ the patient’s current
immunoglobulin therapy infusion usually take place?
Base: Intravenous (160), Subcutaneous (134)
7%
14%
3%
11%
29%
35%
1%
94%
1%
1%
3%
Other
At home
Doctor's surgery/ health clinic
Specialist clinic
Local general hospital
Regional general hospital
Where do infusions take place?
Subcutaneous
Intravenous
In the UK, 40% of intravenous patients are infused at home,
significantly more than intravenous patients on
average
Collected
from
hospital
Delivered Collected
from
pharmacy
Product delivered or collected?
D9b: Is the product delivered, or does it have to be collected? Base: Respondents who receive treatment at home or other place Subcutaneous (127), Intravenous (35)
42% / 20% 35% / 11% 23% / 69%
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10% 9% 21% 21% 39%
Less than 11 mins 11 - 15 16 - 30 31 - 60 60mins+
3% 4% 16% 24% 53%
Less than 31 mins 31-60 61-120 121-180 181mins+
For patients on intravenous administration it takes about an hour on average to get to the place of therapy. It takes 3 and a half hours to carry out the infusion
part of the process. Overall time to complete treatment is over 6 hours.
D10 Approximately, how long does it typically take you to get to the ... where your
current immunoglobulin therapy takes place?
D11 How long does it typically take for ...Pre and Post infusion time (all the other
time spent at the centre but not actually receiving product e.g. waiting to be checked
in, loading the vials etc.)? Carry out the infusion part of the process?
Base: Intravenous (160)
IV patients: Time it takes to…
19% 31% 20% 22% 7%
0 1 - 15 16 - 30 31 - 60 61 - 180 181+Average time
1 hour 6 min
Get to where the immunoglobulin therapy takes place
1.5 hours
Pre and post infusion time (all the other time spent at the centre but not actually receiving product)
Carry out the infusion part of the process
3 hours 46 mins
6 hours 21 mins
Overall time for
IV treatment:
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10% 34% 44% 7% 4%
Less than 31 mins 31-60 61-120 121-180 181mins+
For subcutaneous patients it takes under 20 min on average to set up the infusion equipment and 1.5 hours to complete the infusion part.
The average overall time it takes to complete one session is under 2 hours.
D11 How long does it typically take for
.. Setting up the infusion equipment (to get everything done to the point of injection including
preparation of the injection site)?
…Carry out the infusion part of the process?
Base: Subcutaneous (134)
SubC patients: How long does it take to…
16% 25% 22% 24% 13%
Less than 6 minutes 6 - 10 11 - 15 16 - 30 31+
Average time
19 min
Setting up the infusion equipment (inc. preparation of the injection site)
Carry out the infusion part of the process
1.5 hours
1 hour 41 mins
Overall time for
SubC treatment:
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Almost all intravenous patients use one infusion site at a time. For subcutaneous patients using two sites is the most common (53%),
with a quarter of patients using 3 or more sites during an infusion session.
D12a How many infusion sites do you use at a time?
D12b Has this changed over time?
Base: Intravenous (160), Subcutaneous (134)
96%
2%
1%
1%
21%
53%
11%
12%
3%
1
2
3
4
5+
Infusion sites used at a time
Intravenous
Subcutaneous
5%
9%
78%
72%
17%
19%
Intravenous
Subcutaneous
Decreased Not changed Increased
Has this changed over time? Number of infusion sites used…
1.2
2.3
Average number
of infusion sites
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4% 4%
13%
53%
26% All occasions
Most occasions
Some occasions
Few occasions
Very few occasions
Amongst IV patients, 78% can find a vein on first try on most or all occasions. For those who don’t find a vein for the first time,
it takes an average of three attempts
D14. Which of the following statements best described the ease of
finding a vein when administering immunoglobulin therapy? ‘I/my
care giver can usually find a vein the first time.
D14b. Approximately how many tries does it typically take to find a
vein for intravenous administration?
Base: All Respondents answering about IV therapy (160)
Frequency of finding a vein on first attempt (IV patients)
2; 51% 3; 23%
4; 17% 5+; 9%
3
Average
How many tries does it
typically take to find a vein?
Base: IV respondents who usually find a vein the first time on some / few / very few occasions (35)
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1 in 6 patients who receive subcutaneous therapy use a peristaltic/roller pump, with almost two third using a syringe driver
to administer the infusion
D16: Do you use a peristaltic pump or a syringe driver?
D17: How many ...s' do you use each time you administer immunoglobulin?
Base: Subcutaneous (134)
8%
7%
8%
6%
13%
3%
2%
39%
14%
Don't know
Hand push/ don't use pump
Other size/Unsure
60ml driver
50ml driver
40ml driver
30ml driver
20ml driver
Peristaltic/roller pump
Peristaltic pump or syringe driver (SubC only)
14%
63%
Syringe driver
How many syringe drivers do you
use each time to administer
immunoglobulin?
58%
34%
7%
1 2 3 4
Base: Subcutaneous therapy using a syringe driver (95)
1.5
Average 44% use one and 44% use two drivers each time they administer.
12% use more than two drivers.
94% use one driver each time they administer
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How the time is spent during the treatment very much depends on the type of administration (IV/SubC) and the place of therapy.
As SubC therapy is primarily administered at home, SubC patients can spend time on more home activities
D18: How do you/does the patient spend your/their
time during your/their treatment?
Base. Intravenous (160), Subcutaneous (134)
47%
57%
41%
37%
27%
46%
16%
15%
11%
1%
35%
1%
3%
2%
76%
49%
2%
3%
40%
12%
26%
44%
13%
20%
37%
1%
1%
1%
Watching television / DVDs
Reading
Talking to other patients
Talking to nurse
Talking to friends / family
Sleeping
Playing computer games
Using the internet
Working
Household chores
Eating
Do nothing
Playing with toys
Listening to music/radio
Time spent during treatment
Intravenous
Subcutaneous
As most intravenous patients need to visit a hospital to get
their treatment, a significantly higher share of them spends time with sleeping, talking to
nurses and other patients.
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I. Treatment
Evaluation of current treatment / unmet needs
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Overall, three quarters (74%) are satisfied with their treatment. However, 1 in 5 (18%) are dissatisfied with the number of needles
to contend with each month
D19a: How satisfied are you with the following…
Base: All Respondents (300), SubC only (134), IV only (160)
3%
4%
3%
3%
7%
5%
8%
10%
3%
11%
14%
11%
28%
33%
16%
18%
25%
51%
42%
36%
26%
39%
21%
30%
25%
41%
26%
27%
41%
56%
32%
Overall Satisfaction
Device used to performinfusion
Frequency of sessions
Number of needles permonth
Time taken to set upinfusion (SC only)
Time to get to therapy
Waiting time athospital/clinic (IV only)
Completely dissatisfied Quite dissatisfied Neither/ nor Quite satisfied Completely satisfied
Waitin
g tim
es
Infu
sio
n s
essio
n
Dissatisfied Satisfied
14% 62%
14% 53%
10% 62%
6% 83%
10% 76%
3% 80%
6% 77%
Satisfaction with aspects of current treatment
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Subcutaneous patients like their treatment in all aspects of acceptability; time administration takes, being able to fit treatment into schedule,
ability to self-administer and overall convenience
D19b. Please indicate the extent to which you like or dislike the following aspects
of your current immunoglobulin therapy ...
Base: Intravenous (160), Subcutaneous (134)
10%
6%
18%
4%
26%
18%
13%
3%
13%
3%
14%
4%
26%
16%
23%
6%
39%
5%
22%
7%
27%
39%
27%
17%
12%
17%
35%
33%
12%
26%
31%
73%
19%
74%
25%
55%
Intravenous
Subcutaneous
Intravenous
Subcutaneous
Intravenous
Subcutaneous
Intravenous
Subcutaneous
Dislike very much Dislike No preference Like Like very much
Acceptability of aspects of current Ig treatment
Time administration
takes
Being able to fit treatment into
schedule
Ability to self-administer
The overall convenience
Dislike Like
36% 39%
19% 65%
19% 58%
4% 90%
30% 31%
4% 91%
18% 60%
5% 88%
Significant difference between intravenous and subcutaneous therapy:
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Subcutaneous therapy is perceived to perform better on a number of aspects relating to quality of life (convenience, allowing independence and personal
freedom) in the survey sample
D19c. And would you say your/the patient’s current immunoglobulin therapy is…?
Base: All Respondents (300)
Inconvenient
Painful
Have not improved health
Interferes with social/family life
Interferes with work / school
Given in place where uncomfortable
Requires too much time waiting before
Given in unpleasant atmosphere
Waste of time
Make me anxious or nervous
Seems to be too expensive
Makes me too dependent
Requires lot of travel time & cost
Doesn't limit freedom to take trips / move
Not scheduled to convenience
Convenient
Not at all painful
Have improved health
Don't interfere with social/family life
Don't interfere with work/school
Given in place where comfortable
Don't require too much time waiting before
Given in pleasant atmosphere
Worthwhile
Don't make me anxious or nervous
Don't seem to be too expensive
Don't make me too dependent
Require v. little travel time and cost
Limits freedom to take trips or move
Scheduled according to convenience
0 1 2 3 4 5 6 7
Intraveneous Subcutaneous
Significant difference between intravenous and subcutaneous therapy:
Evaluation of current therapy in terms of quality of life (mean scores)
performing better
-
More subcutaneous patients experience side effects at the infusion site compared to IV patients. However, for the majority these side effects have
low or medium impact on their life and they do not seek to change therapy.
3%
4%
2%
3%
1%
4%
4%
8%
30%
11%
28%
4%
15%
4%
17%
17%
58%
28%
40%
16%
30%
14%
31%
72%
5%
56%
25%
79%
46%
77%
45%
Intravenous
Subcutaneous
Intravenous
Subcutaneous
Intravenous
Subcutaneous
Intravenous
Subcutaneous
Large impact on life/discussed with doctor/Switching Large impact on life/Discussed with doctor/Not switching
Large impact on life/Not discussed with doctor Medium impact on life/Not looking to change
Low impact/ Not seeking to change Not experiencing this
To what extent is life impacted by current therapy – side effects at infusion site
Swellings/bumps at infusion site
Pain at infusion site
Itching at infusion site
Hardness at infusion site
D19d. Few treatments are perfect, and so considering the list of possible treatment-
related side effects, please indicate how much your life is currently impacted by each,
on your current medication
Base: Intravenous (160), Subcutaneous (134)
Large Impact Experiencing
side effect
4% 28%
7% 95%
5% 44%
5% 75%
2% 21%
9% 54%
5% 23%
7% 55%
Significant difference between intravenous and subcutaneous therapy:
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More IV patients experience headaches as a side effect of the therapy. 1 in 10 IV patients discussed switching with their doctors because of headaches, but only 2% is moving to another treatment as a result.
4%
7%
8%
3%
9%
5%
3%
3%
15%
9%
14%
6%
24%
14%
14%
12%
49%
69%
55%
67%
Intravenous
Subcutaneous
Intravenous
Subcutaneous
Large impact on life/discussed with doctor/Switching Large impact on life/Discussed with doctor/Not switching
Large impact on life/Not discussed with doctor Medium impact on life/Not looking to change
Low impact/ Not seeking to change Not experiencing this
To what extent is life impacted by current therapy – side effects at infusion site
Headaches
Other side effects
D19d. Few treatments are perfect, and so considering the list of possible
treatment-related side effects, please indicate how much your life is currently
impacted by each, on your current medication
Base: Intravenous (160), Subcutaneous (134)
Large Impact Experiencing
side effect
11% 51%
7% 31%
16% 45%
14% 33%
Significant difference between intravenous and subcutaneous therapy:
-
1 in 3 (35%) intravenous patients consider it important to reduce the occurrence of headaches as a side effect of the intravenous Ig therapy
D19e. How important to you would it be to improve on…?
Base: Intravenous (160)
46% 36%
51% 59%
9%
9%
13% 8%
9%
11%
8% 9% 12%
9%
11% 9% 8%
8%
6% 5%
16%
27%
11% 11%
Pain at infusion site Headaches Swelling/bumps at infusionsite
Itching at infusion site
Highly important (5)
4
3
2
1
Highly unimportant (0)
Importance of improving on aspects of treatment – Intravenous administration
Important (4-5) 23% 34% 18% 16%
Unimportant (0-1) 56% 45% 63% 66%
Intravenous
44% experience 51% experience 28% experience 21% experience
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Pain and swelling at the infusion site are considered to be important areas to improve by subcutaneous patients. Headaches affect less patients, but for
those affected it is an important area to improve
D19e: How important to you would it be to improve on…?
Base: Subcutaneous (134)
19%
53%
13%
36%
16%
12%
19%
16% 15%
4%
16%
12% 15%
6%
19%
13% 19%
6% 18% 11%
16% 19% 13% 12%
Pain at infusion site Headaches Swelling/bumps at infusionsite
Itching at infusion site
Highly important (5)
4
3
2
1
Highly unimportant (0)
Importance of improving on aspects of treatment – Subcutaneous administration
Important (4-5) 35% 25% 31% 23%
Unimportant (0-1) 35% 65% 33% 51%
Subcutaneous
75% experience 31% experience 95% experience 54% experience
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II. Living with PID
-
84% of all patients with PID visit a specialist immunology doctor in relation to their PID.
D23a. Which of the following specialist doctor(s) do you/ the patient recently
visit relating to your/their PID?
Base: All Respondents (300)
84%
14%
14%
12%
6%
6%
5%
5%
4%
4%
3%
2%
2%
1%
82%
6%
2%
1%
4%
1%
1%
1%
Specialist Immunology Doctor
Infectious Disease specialist
Pulmonary specialist
Gastroenterologist
Oncologist
ENT specialist
Paediatrician
Hematologist
Rheumatologist
GP
Allergy specialist
Neurologist
Dermatologist
Ophtalmologist
Doctor visited in relation to PID
Visited most regularly
Doctor visited in relation to PID
In the UK and Canada a significantly higher share of
patients (97% and 94% respectively) visits an immunology
specialists in relation to PID
-
55%
16%
11%
8%
1%
8%
0
1
2
3
4
5
Subcutaneous
45% of subcutaneous patients delayed self-administering by three or more days in the last six months at least once, but 82% haven’t skipped a dose.
Among IV patients 9 in 10 haven’t skipped a dose, significantly more than for SubC.
D20b Thinking about your usage of immunoglobulin, how many times in the past six
months do you estimate you have… Base: Intravenous (160), Subcutaneous (134)
Delayed self-administering by 3 days or more Skipped a dose
Number of times in the last 6 months
92%
5%
3%
1%
82%
10%
4%
4%
0
1
2
3+
IntravenousSubcutaneous
Number of times in the last 6 months
Significant difference between intravenous and subcutaneous therapy:
-
Sixty-six percent (using intravenous Ig) and 70% (using subcutaneous Ig) report missing 10 or fewer work/school days during the past 6 months. Of these, 35%
(using intravenous Ig) and 37% (using subcutaneous Ig) missed 0 days
H4: In the last 12 months, how many unscheduled or
emergency visits have you/the patient
made to each of the following in relation to PID?
H5: And how many days have you/the patient missed
at work/education due to ill health in
the past six months?
Base: All Respondents (300)
Regional general
hospital
Local general
hospital Specialist clinic
Doctor’s surgery/
health centre
Ave: 1.2 Ave: 1.0 Ave: 1.3 Ave: 3.6
Unscheduled visits in relation to PID in last 12 months
Days missed at work/education due
to ill health in last 6 months
There was no difference in
the number or unscheduled
visits based on the type of
administration (IV vs. SubC)
72%
7%
7%
4%
3%
9%
0
1
2
3
4
5+
72%
8%
8%
3%
4%
6%
0
1
2
3
4
5+
69%
7%
9%
4%
4%
8%
0
1
2
3
4
5+
43%
9%
11%
6%
6%
25%
0
1
2
3
4
5+
35%
31%
8%
9%
5%
13%
37%
33%
11%
3%
8%
8%
0
Less than 11
11 - 15
16 - 30
31 - 60
61+
Intravenous
Subcutaneous
-
PID patients would like to take part in ‘everyday’ activities: travelling / going abroad was mentioned by most (19%) of subcutaneous patients as the one thing they would like to be able to do, but don’t feel they can because of PID
H6: In your opinion, what is the one thing you would LIKE to be able to do but don't
feel you can, because of PID? MULTIPLE RESPONSE
Base: All Respondents (300)
16%
14%
12%
6%
8%
6%
10%
6%
3%
5%
4%
4%
2%
1%
2%
1%
1%
19%
20%
14%
10%
6%
7%
4%
6%
9%
6%
7%
2%
1%
4%
2%
1%
2%
Travel abroad
More energy/not be tired all the time
Be able to exercise more
Relax, not anxious about infection
Get back to work
Extended holidays
Lead normal life/not take medication
Go to the swimming pool
Have a social life
Go to crowded places
Feel well/not live with restrictions
Not have pain/feel sick
Get back to the life I used to have…
Be with sick people
Do the job I want
Travel by plane
Eat what I want
Intravenous
Subcutaneous
“Have enough energy for a full day and going out at night without needing a nap and early night!”
“Travel for over 2 weeks without the need to bring medical
equipment or make advanced planning.”
“Travel to countries very far away.”
“Get back to life I used to have before I was diagnosed with PID.”
“I would like to meet other people even during times there's a lot of flu
etc. Don't like trying to avoid crowded places etc. because the
risk for infection.”
One thing patient would LIKE to be able to do but don’t feel they can, because of PID (spontaneous mentions)
-
Outlook on life with PID over next five years
H7: Generally speaking, how optimistic is your outlook on life, living with
PID in the next 5years?
Base: All Respondents (300), Intravenous (160), Subcutaneous (134)
9%
11%
5%
25%
30%
19%
41%
30%
54%
23%
27%
20%
All respondents
Intravenous patients
Subcutaneous patients
Very pessimistic Quite pessimistic Neither optimistic nor pessimistic Quite optimistic Very optimistic
More subcutaneous patients are optimistic about their lives over the next five years than intravenous patients
Pessimisti
c
Optimisti
c
10% 65%
13% 57%
6% 75%
Significant difference between intravenous and subcutaneous therapy:
-
III. Future Treatment Needs
Ideal treatments and conjoint analysis
-
Intravenous and subcutaneous patients differ regarding the features they look for in an ideal product to treat PID.
D21: Thinking about future PID treatments imagine you were working with a medical
design team what two features would you look for in the ideal product?
Base: All Respondents (300), Intravenous (160), Subcutaneous (134)
30%
30%
26%
18%
16%
12%
10%
9%
7%
2%
2%
21%
19%
37%
4%
25%
7%
9%
6%
10%
9%
10%
Fewer/no side effects
Shorter administration time
Therapy with tablets/patches/stop using needles
Ability to administer at home
Longer time between infusions
More efficacy/cures the problem completely
Ease of administration/easy to use
Safe product
Less painful
Preloaded syringes/ product ready tobe used
No need to be refrigerated/easy to store/transport
Intravenous
Subcutaneous
Compared to subcutaneous patients, a significantly higher share of intravenous patients
mentioned ‘shorter administration time’ and ‘ability
to administer at home’ as a feature of an ideal product.
Among subcutaneous patients ‘longer time between infusions’
features more often as an attribute of an ideal product. 9%
of them would like to use ‘preloaded syringes’.
Features of an ideal treatment for PID (spontaneous mentions)
Significant difference between intravenous and subcutaneous therapy:
-
Amongst both intravenous and subcutaneous patients, the positive elements of their current treatment (e.g. intravenous – less frequent infusions needed) seem to have more weight in the decision about how therapy is administered
Importance of attributes by current route of administration (intravenous vs. subcutaneous)
Conjoint analysis
Base: All Respondents (300)
All Respondents
Current
Intravenous
Current
Subcutaneous
Convenience around scheduling
15% 13% 18%
Dosing frequency 19% 24% 14%
Where you take the treatment
22% 17% 29%
Number of needle sticks per treatment
20% 24% 15%
Time to take each treatment 23% 23% 23%
High
Average to high
Average
Low
Colour coding
The colour coding
indicates whether
a score of the
subgroup is higher
or lower compared
to the score at
total level.
-
Utility shares show how appealing each level is, compared to other levels within the attribute. The larger the share of a level, the higher its appeal.
65%
35%
Self-administer
Appointment with HC professional
Convenience around scheduling
68%
18%
15%
Once a month
Twice a month
Weekly
Dosing frequency
80%
20%
Home
DRs office / surgery, hospital
Where you take the treatment
63%
27%
10%
1
2
3
Number of needle sticks per treatment
70%
24%
6%
2 hours
4 hours
6 hours
Time to take each treatment
Base: All Respondents (300)
Note that the utility share of levels of belonging to different attributes cannot be compared.
Each block adds up to 100% (as they are shares).
Conjoint analysis
-
IV. General Health
EQ-5d, SF12-v2, SF10 (© 1990 EuroQol Group. EQ-5D™ is a trade mark of the EuroQol Group)
-
EQ5d: Please indicate which statements best describe your/the patient’s health today
Base: All Patients (216)
Weighted Summary Index (using UK preference weights, TTO value set)
0,683 0,666 0,674
0,79
0,62 0,65
0,77 0,73
0,68 0,71
0,78
0,71 0,67
0,0
0,1
0,2
0,3
0,4
0,5
0,6
0,7
0,8
0,9
1,0
EQ-5d– Weighted Summary Index
Patient health – EQ-5d (© 1990 EuroQol Group. EQ-5D™ is a trade mark of the EuroQol Group)
NOTE: PATIENTS ONLY
Better health
Worse health
EQ-5d
-
PID patients are below US norms across Physical and Mental elements
SF-12 ®
Health Survey
F1. In general, would you say your health is… F2. Does your health now limit you in these activities. F3: During the past 4 weeks, how much of the
time have you had any of the following problems with your work/other regular daily activities as a result of your physical health? F4 During the past 4
weeks, how much of the time have you had any of the following problems with your work or other regular daily activities as a result of any emotional
problems (such as feeling depressed or anxious)? F5: During the past 4 weeks, how much did pain interfere with your normal work (including both
work outside the home and housework?)
F6: How much time during the past 4 weeks … F7 During the past 4 weeks, how much of the time has your physical health or emotional problems
interfered with your social activities (like visiting friends, relatives, etc.)? Base: All Respondents who are PID patients (216)
40,7
46,0 42,4 41,1
44,3
37,9
45,2 41,7
44,2 46,5
0
10
20
30
40
50
60
70
80
90
100
PhysicalComponent
Summary (PCS)
MentalComponent
Summary (MCS)
PhysicalFunctioning
(PF)
Role-Physical(RP)
Bodily Pain (BP) General Health(GH)
Vitality (VT) SocialFunctioning (SF)
Role-Emotional(RE)
Mental Health(MH)
SF-12 Component Scores – Norm Based Scores (NBS) Scores for Total Sample
US norm
50
Better
health
Worse
health
Physical Health Scores Mental Health Scores
SF-12v2® Health
Survey
-
SF – 10 – Summary Scores
G1: In general, would you say your child’s health is… G2: During the past 4 weeks, has your child been limited in any of the following activities due to
HEALTH problems? Bending, lifting or stooping / Doing things that take some energy such as riding a bike or skating G3: During the past 4 weeks, has
your child been limited in the KIND of schoolwork or activities with friends he/she could do because of PHYSICAL health problems? G4: During the past 4
weeks, has your child been limited in the KIND of schoolwork or activities with friends he/she could do because of EMOTIONAL or BEHAVIOURAL
problems? G5: During the past 4 weeks, how much bodily pain or discomfort has your child had? G6: During the past 4 weeks, how satisfied do you think
your child has felt about his/her friendships? G7: During the past 4 weeks, how satisfied do you think your child has felt about his/her life overall? G8:
During the past 4 weeks, how much of the time do you think your child behaved as if he/she was bothered or upset? G9: Compared to other children your
child’s age, in general would you say his/her behaviour is? Base: All Respondents who are carers of PID patients who are 50
Living with PID has a substantial impact on the physical wellbeing of the children. Psychosocially they are less affected, with their score being only slightly below the US norm.
36,9
48,1
0
10
20
30
40
50
60
70
80
90
100
Physical (PHS) Psychosocial (PSS)
US norm
50
SF-10 for
Children™
-
V. Information Sources
-
28% of people have used the IPOPI website to source information about PID over the past 12 months, with 69% of them finding it “very useful”.
35% expect to use it in the next 12 months
D24a: Which of the following sources, if any, have you used to find out information about PID in the past 12 months,
and which do you expect to use over the next coming months? Base: All Respondents (300)
D24b: You indicated that you have used the following sources to find information on PID over the past 12 months.
How useful were they? Base: Those who have used the source to find information about PID in the past 12 months
79%
45%
22%
44%
31%
24%
10%
28%
34%
21%
23%
12%
77%
74%
21%
1%
50%
30%
15%
36%
30%
23%
22%
35%
29%
23%
25%
23%
51%
47%
30%
5%
Specialist Immunology Doctor
Nurse
Family and friends
Meetings / conferences / events
Books, journals etc
Newspapers or magazines
Television or radio
IPOPI website
Internet blogs or forums
Social networking sites (e.g. Facebook)
Pharmaceutical company websites
Government/ local authority website
NMO website
Other NMO information
Other IPOPI information
None of the above
Have used in last 12 months Expect to use in next 12 months
Sources of information regarding PID
28% of people have used the IPOPI website to source
information about PID over the past 12 months, with 69% of them finding it “very useful”.
35% expect to use it in the next 12 months
% of users who found source ‘very useful’
83%
73%
28%
75%
51%
11%
10%
69%
32%
14%
36%
26%
76%
77%
71%
-
The most widely used NMO services used by members are therapy information and health / social support
H8: Which of the following does provide you (the
patient/the carer) in terms of support?
Base: All Respondents (300)
19%
21%
34%
41%
61%
40%
36%
35%
37%
21%
41%
43%
31%
22%
17%
Guidance on eco.support
Travel info.
Info. about product differences
Health/social support
Info. about therapy
Provided and used Provided but not used Not provided NET: Provided
82%
78%
69%
57%
59%
NMO services provided and used by Members
-
17% of the patients are happy with the support provided by their national patient organisation as it currently is, but suggestions to improve support
were made as shown below
H9 How could better service the needs of the PID community in ...? MULTIPLE RESPONSE
Base: All Respondents (300)
17%
12%
9%
9%
8%
7%
6%
5%
5%
4%
4%
18%
Happy with the current situation
Have more and better information about PID
Regional meetings for patients to meet each other
Hospital personnel/Doctor more or better trained on PID
More advertising
Financial support
Have more regional offices
Better website
Keeping patients up to date with everything that affects PIDcommunity
Pressure official organisations to recognise the disease
More support
Don't know
What can the NMO do to better serve the needs of the PID community? (spontaneous answers)
-
VI. Sample profile
-
7 in 10 survey respondents are patients with the remaining made up by care givers. CVID is the most widely represented diagnosis of PID.
P1: Which of the following best describes your role In relation to PID?
D1: And what is your specific diagnosis/ What is the diagnosis of the PID patient you care for?
Q.C0b/Q.C3: Are you ... Male or Female
Base: All Respondents (300)
72%
28%
Role in relation to PID
PID patient
Care-giver
2%
8%
1%
2%
3%
9%
12%
13%
51%
0% 10% 20% 30% 40% 50% 60% 70% 80% 90% 100%
Don’t know
Other
Wiskott-Aldrich Syndrome
Selective IgA Deficiency
Hyper-IgM Syndrome
IgG Subclass Deficiency
X-Linked Agammaglobulinemia (XLA)
Hypogammaglobulinemia
Common Variable Immunodeficiency(CVID)
Patient Diagnosis
48%
48%
49%
52%
52%
51%
All respondents
Intravenous
Suncutaneous
Male Female
Gender
-
Almost half (46%) of patients experienced a significant delay between their initial suspicion of PID and a formal diagnosis
C2b: To the best of your knowledge, was there a delay between the initial
suspicion of PID and its formal diagnosis?
Base: All Respondents (300)
8%
7%
10%
22%
28%
17%
24%
24%
23%
46%
42%
50%
All respondents
Intravenous
Suncutaneous
Don't know No Yes - minor delay Yes - significant delay
Delay in official PID diagnosis
-
15%
17%
10%
16%
14%
14%
12%
2%
1-10 years old
11-20 years old
21-30 years old
31-40 years old
41-50 years old
51-60 years old
61+
Prefer not to say
Age of patient
The age profile of intravenous respondents is in line with those on subcutaneous treatment for each age bracket,
except for the 51-60 year-olds
Q.C0a/Q.C1 How old are you?
C2: How old were you when first diagnosed with PID?
Base: All Respondents (300)
6%
32%
10%
13%
17%
9%
7%
4%
1%
1%
Less than 1 year
1 - 10 years
11 - 20 years
21 -30 years
31 - 40 years
41 - 50 years
51 - 60 years
61+
Prefer not to say
No answer
Age at diagnosis
-
Summary of select findings – view of current treatments
IV and SubC treatments are roughly split 50/50 across the
surveyed countries
Impact of treatment
Side effects in IV and SC patients
Immunology Specialist physicians are the decision makers choosing administration route in 2 in 5 cases with patients and or caregivers being secondary influencers.
95% of SubC experience swelling / bumps at infusion site but this causes a large impact for just 7%. 7% experience pain at infusion sites but to a large extent for just 5%. Headaches are most prominent for IV patients (51% experience) and these have a large impact on life for 11%
One in five IV patients have tried SubC whilst
8 in 10 SubC patients have tried IV
IV patients who had tried SubC were most likely to have stopped because of side effects (61%) and wanting longer between doses (32%). SubC patients who had previously tried IV most commonly stopped because of inconvenience of going to the centre (51%), wanting to treat at home (43%) or because it was recommended they change (48%). 33% said side effects
Sixty-six percent of patients (using intravenous Ig) and 70% (using subcutaneous Ig) report missing 10 or fewer work/school days during the past 6 months. Of these, 35% (using intravenous Ig) and 37% (using subcutaneous Ig) missed 0 days. While 39% report no pain, 50% report moderate pain; 48% report problems with daily activities. Patients’ mental well-being is affected too: 61% report no anxiety/depression, while 39% identify moderate or extreme anxiety/depression (averages were below population norms across physical and mental QoL elements)
-
Summary of select findings – unmet needs and drivers of choice
Main area where IV & SC patients would like
treatments to be improved
IV & SC patients views on ideal treatment
A preference was shown for self-administration at
home, infrequent, quick to administer dosing and
few needle sticks
34% of IV patients believe headaches are the most important area to improve. For SubC patients, pain at the infusion site and swelling at the infusion site are priorities for improvement though 19% say it is ‘highly important’ to focus on headaches too.
Spontaneously 30% of IV patients said they wanted a treatment with less side effects compared to 21% of SubC patients. SubC patients were most likely to say a therapy without needles (37%) and more time between infusions (25%). IV patients also mentioned a shorter admin time (30%) and the ability to administer at home (18%)
Half of IV respondents (52%) would prefer a SubC type therapy similar to those available. This would be preferable for 91% of existing SubC respondents.
Preference analysis showed relatively level importance
around attributes in choosing treatments
Drivers of choice were time to take each treatment (23%), site of treatment - at home vs. medical centre (22%), number of needle sticks (20%), dosing frequency (19%) and convenience on scheduling (15%).